林业发展局批准了Yuviwel,这是对患有亚沙伏拉西亚病的儿童每周一次的治疗,以改善生长。 The FDA approved Yuviwel, a weekly treatment for children with achondroplasia, to improve growth.

林业发展局批准了Ascendis Pharma's Pharmas Yuviwel(Navepegritide),这是为2岁及2岁以上的儿童首次每星期治疗一次的Achondroplasia,这是一种罕见的遗传病,造成侏儒症。 The FDA has approved Ascendis Pharma’s Yuviwel (navepegritide), the first once-weekly treatment for children aged 2 and older with achondroplasia, a rare genetic disorder causing dwarfism. 根据三项临床试验,包括一项关键的第三阶段研究,加速批准显示,与安慰剂相比,生长速度和其他关键结果大大改善,其安全情况类似于安慰剂。 The accelerated approval, based on three clinical trials including a pivotal Phase III study, showed significantly improved growth velocity and other key outcomes compared to placebo, with a safety profile similar to placebo. 抗过活性FGFR3信号的该药物预计将在2026年第二季度在美国提供。 The drug, which counteracts overactive FGFR3 signaling, is expected to be available in the U.S. in the second quarter of 2026. 批准中包括罕见的儿科疾病优先审查凭单。 The approval includes a rare pediatric disease priority review voucher.